Scientific advice and protocol assistance

The European Medicines Agency (EMA) can give scientific advice and protocol assistance to medicine developers. For human medicines, scientific advice and protocol assistance are given by the Committee for Medicinal Products for Human Use (CHMP) on the recommendation of the Scientific Advice Working Party (SAWP).

Scientific advice is when the Agency gives advice to a developer on the appropriate tests and studies in the development of a medicine. This is designed to facilitate the development and availability of high-quality, effective and acceptably safe medicines, for the benefit of patients.

Medicine developers can request scientific advice from the EMA at any stage of development of a medicine, whether the medicine is eligible for the centralised authorisation procedure or not.

Scientific advice helps to ensure that developers perform the appropriate tests and studies, so that no major objections regarding the design of the tests are likely to be raised during evaluation of the marketing-authorisation application. Such major objections can significantly delay the marketing of a product, and, in certain cases, may result in refusal of the marketing authorisation. Following the Agency’s advice increases the probability of a positive outcome.

The Agency gives scientific advice by answering questions posed by medicine developers. The advice is given in the light of the current scientific knowledge, based on the documentation provided by the medicine developer.

Scientific advice is prospective in nature. It focuses on development strategies rather than pre-evaluation of data to support a marketing-authorisation application.

Scientific advice received from the Agency is not legally binding on the Agency or on the medicine developer with regard to any future marketing-authorisation applications for the medicine concerned.

Protocol assistance

Protocol assistance is the special form of scientific advice available for developers of designated orphan medicines for rare diseases.

In addition to scientific advice, developers of orphan medicines can receive answers to questions relating to the criteria for authorisation of an orphan medicine. These include:

the demonstration of significant benefit within the scope of the designated orphan indication;

similarity or clinical superiority over other medicines. This is relevant if other orphan medicinal products exist that might be similar to the product concerned and which have market exclusivity in the same indication.

Requesting scientific advice

Medicine developers can request scientific advice or protocol assistance either during the initial development of a medicinal product before submission of a marketing-authorisation application or later on, during the post-authorisation phase.

there appears to be no or insufficient relevant detail in European Union guidelines or guidance documents, or in Pharmacopoeia monographs, including draft documents or monographs released for consultation;

the developer chooses to deviate from the available guidance in its development plan.

Scientific advice on post-authorisation safety studies (PASS)

EMA encourages medicine developers to seek scientific advice for PASS protocols. This voluntary, optional procedure will help to improve the design of studies meant to collect further information on a medicine's safety once it is on the market.

Parallel consultations from regulators and HTA bodies

EMA offers consultations in parallel with European Network for Health Technology Assessment (EUnetHTA) as of July 2017. This aims to allow medicine developers to obtain feedback from regulators and HTA bodies on their evidence-generation plans to support decision-making on marketing authorisation and reimbursement of new medicines at the same time.

The procedure is a single gateway for parallel consultations with EMA, EUnetHTA and HTA bodies on their evidence-generation plans.

Consultations can take place before or after the product is made available on the market. The objective is to help generate optimal and robust evidence that satisfies the needs of both regulators and HTA bodies.

Tailored scientific advice pilot on biosimilars

EMA launched a tailored scientific advice pilot project in February 2017 to support the development of new biosimilars.

The tailored procedure advises developers on the studies they should conduct, based on a review of the quality, analytical and functional data they already have available.

The pilot is open to all types of biosimilars and includes a pre-submission meeting to review the suitability of the data package. Applicants should note that the SAWP will need an extra month in addition to normal scientific advice timelines to review applications.

EMA plans to run the pilot until it has completed six scientific advice requests, with maximum one scientific advice request accepted per month. The Agency will analyse the outcome after completing the pilot.